Welcome to Gene Therapy Net

Gene Therapy Net is the web resource for patients and professionals interested in gene therapy. The objectives of Gene Therapy Net are to be the information resource for basic and clinical research in gene therapy, cell therapy, and genetic vaccines, and to serve as a network in the exchange of information and news related to above areas. In addition, Gene Therapy Net provides an overview for sponsors and researchers of the different international regulations and guidelines associated with clinical gene therapy trials. For resources pertaining to regenerative cell therapy and clinical treatment, check out R3 Stem Cell.

Gene Therapy in Mesothelioma Patients

Posted on: 28 September 2020, source: mesothelioma.com
Around 1 in 16 people will be diagnosed with lung cancer in their lifetime; this accounts for about 13% of all new cancer diagnoses. However, even with a relatively lower diagnosis rate, lung cancer currently has the highest number of cancer deaths at nearly 22%. Among all the various subtypes of cancer categorized under the umbrella term, “lung cancer,” mesothelioma is one of the most lethal forms.
Despite accounting for around .2% of all cancer diagnoses in the United States, mesothelioma’s lethality is among the highest of all forms of cancer. Pleural mesothelioma, the most common form of this cancer, has a 10-year survival rate of only about 5%. Currently, the only known cause of mesothelioma is asbestos exposure, and although no amount of exposure is safe, it is important to note that your risk of developing mesothelioma drastically increases the longer you are exposed. In addition, due to a prolonged latency period, many patients are diagnosed far too late for proper treatment — usually around the age of 65-74.

FDA grants orphan drug designation for LHON gene therapy

Posted on: 26 September 2020, source: FDA
Neurophth Therapeutics has received orphan drug designation from the FDA for NR082 for the treatment of Leber hereditary optic neuropathy associated with ND4 mutation, according to a press release. Between 70% and 90% of LHON cases are caused by ND4 mutation, according to the release.

New HIV Gene Therapy, CAR-T Treatments Could be on the Horizon for Patients

Posted on: 8 September 2020, source: BioSpace
The National Institutes of Health (NIH) has backed researchers at the University of Southern California and the Fred Hutchison Cancer Center with a five-year, $14.6 million grant to develop a gene therapy that could potentially control HIV without the need for daily medications. Most HIV patients take a well-regimented cocktail of medications each day to control the virus. This therapy could change that.

Study Confirms Efficacy of Gene Therapy for Spinal Muscular Atrophy in Children

Posted on: 30 August 2020, source: Technology Networks
In May 2019, the U.S. Food and Drug Administration (FDA) approved a gene replacement therapy for the inherited, progressive neuromuscular disease 5q-linked spinal muscular atrophy (SMA). Approval included all children with SMA under the age of two years; however, the gene therapy had only been studied in children aged up to 8 months. Now, a new study discusses safety and early outcomes in a large cohort of SMA patients under the age of two years who were treated with gene therapy. The report is published in the journal Pediatrics.

FDA Approves First Cell-Based Gene Therapy For Adult Patients with Relapsed or Refractory MCL

Posted on: 4 August 2020, source: FDA
The U.S. Food and Drug Administration approved Tecartus (brexucabtagene autoleucel), a cell-based gene therapy for treatment of adult patients diagnosed with mantle cell lymphoma (MCL) who have not responded to or who have relapsed following other kinds of treatment. Tecartus, a chimeric antigen receptor (CAR) T cell therapy, is the first cell-based gene therapy approved by the FDA for the treatment of MCL. “Tremendous progress has been made in the discovery of new therapies for debilitating diseases that are difficult to treat. This approval is yet another example of customized treatments that use a patient’s own immune system to help fight cancer, while using a scientific advance in this promising new area of medicine,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research. “We’re seeing continued advances in the field of gene therapy and remain committed to supporting innovation in this promising new area of medicine.”

Two deaths in gene therapy trial for rare muscle disease

Posted on: 30 June 2020, source: ScienceMag
Two boys have died after receiving high doses of a gene therapy treatment for their rare muscle disease, Biopharma Dive reports. The patients, born with x-linked myotubular myopathy, developed liver problems that apparently led to sepsis, according to a 23 June letter to patient groups from trial sponsor Audentes Therapeutics. They were older patients and had existing liver disease; several younger patients who got lower doses of the treatment have done well and now breathe on their own without a ventilator.

Preclinical Study of SLS-004, Potential Parkinson’s Gene Therapy, Starting

Posted on: 3 June 2020, source: Parkinson News
Seelos Therapeutics announced it is beginning, earlier than expected, a preclinical study into its investigational gene therapy candidate for Parkinson’s disease called SLS-004. Nerve cell damage in Parkinson’s is caused by the buildup of toxic forms of the alpha-synuclein protein, and resulting clumps of misfolded proteins known as Lewy bodies. These toxic aggregates damage and eventually kill nerve cells — called dopaminergic neurons — in a region of the brain that regulates muscle movement and coordination.